Nanoscope Therapeutics Inc., a company based in Bedford, Texas, has reported vision improvements for advanced retinitis pigmentosa (RP) patients after one year following a single intravitreal injection in a phase 1/2a clinical study. According to the company, the treatment uses a polychromatic opsin to activate ambient light with a single intravitreal injection of “vMCO-010” or “MCO-therapy”. The optogenetics gene therapy strategy turns bipolar cells into light-sensing activated neurons in response to light, making them the new photoreceptors of the retina. The strategy develops an optogenetic gene therapy using “multi-characteristic opsin” (MCO) to re-sensitize the retina to detect low light levels. CEO Sulagna Bhattacharya of Nanoscope has now proposed that “we expect to begin the first randomized, placebo-controlled, double-masked Phase 2b multi-center optogenetic trial in the US this summer to further validate our gene therapy’s ability to improve clinically meaningful vision in RP patients”.
In the study, three patients received low dose (1.75 × 1011 VG per eye) and eight received a high dose (3.5 × 1011 VG per eye). Reporting from the company, florescence imaging of the retina revealed successful gene transduction. At completion, 6 out of 7 (86%) high dose MCO-therapy subjects gained >0.3 logMAR (15 letters). The company reported that all subjects had objective and subjective improvement in functional vision and commented that “the shape discrimination accuracy improved to >90% in all subjects compared to baseline. Further, the performance in two different mobility tests improved by 50% reduction in time to touch lighted panel. These test outcomes were highly correlated with improved patient reported outcomes”. The company’s pipeline includes vision restoration in patients with retinitis pigmentosa, Stargardt disease, and age-related macular degeneration.
Nanoscope received orphan drug designation from the US Food and Drug Administration (FDA), using a proprietary AAV2 vector to deliver the MCO genes into the retina. This mutation-independent gene therapy involves a single injection through the eye administered in a doctor’s office. Samarendra Mohanty, Ph.D., Nanoscope’s President, Chief Scientific Officer and inventor of the technology, said, “optogenetics is a powerful research tool, but had limited scope of clinical benefit because the opsins had a narrow band of activation, unlike natural light environment. MCO is sensitive to broadband light and activatable by ambient light, thus eliminating the risk of photo-toxicity from long-term continuous use of external intense light stimulation devices.”